Presentation (CTI)

OBJECTIVES: Gaucher disease is a rare LSD, which is responsible for a significant clinical, economic, and quality-of-life burden. In India, the limited availability of disease-specific therapies such as ERT and SRT is due to high treatment costs and limited healthcare resources. There is little economic data in India that contrasts these therapies in the Indian health care context. The objective of this study was to assess the cost-effectiveness of ERT and SRT in the Indian healthcare system to inform evidence-based decision-making and policy development for rare diseases such as Gaucher disease.
METHODS: In India, the cost-effectiveness of ERT and SRT can be assessed in patients with Gaucher disease using a comprehensive HTA framework. A systematic literature review of published evidence worldwide was conducted to identify data on clinical effectiveness, safety, treatment outcomes, utility, and disease progression. The direct medical costs, such as drug costs, hospitalization, diagnostics, monitoring, and supportive care, were estimated from primary sources of Indian health care costs and published literature.
RESULTS: The clinical effectiveness analysis showed that both ERT and SRT had beneficial effects on clinical outcomes and quality-adjusted survival compared with supportive care, but there were substantial differences in treatment costs and long-term economic outcomes. ERT was associated with higher overall treatment costs because it requires lifelong infusion therapy, whereas SRT showed potential economic benefits in certain patient populations due to reduced administration and monitoring burden.
CONCLUSIONS: The study provides economic evidence on benefits of ERT and SRT in the Indian context of Gaucher disease. The results demonstrate the difficulty in funding high-cost rare disease treatments in a resource-limited healthcare system and the need for value-based pricing, access policies, and strong HTA systems for rare diseases. Future studies that include patient outcomes and real-world data from India are critical to further support reimbursement/policy decisions for management of Gaucher disease.